Prepared by Queta Bond & Elaine Gallin QE Philanthropic Advisors December 2011

1. Partnering with Patient Advocacy Groups &Voluntary Health Organizations Can Bridge Gaps in Clinical Research Prepared by Queta Bond & Elaine Gallin QE Philanthropic Advisors December 2011

2. BACKGROUND The Clinical Research Forum (CRForum) is a non-profit organization that comprises academic health centers, professional societies, and industry. CRForum’s mission is to provide leadership to the national clinical & translational research enterprise and promote understanding and support for clinical research and its impact on health and health care. www.clinicalresearchforum.org

3. BACKGROUND • CRForum has identified enhancing collaborations between academic health centers (AHCs) and patient advocacy groups/voluntary health organizations (PAG/VHOs) as a priority. • The goal of the Partnering with PAG/VHO Initiative is toenhance translational and clinical research collaborations between AHCs & PAG/VHOs. • Steering Committee members: Bill Crowley, Pamela Davis, Richard Galbraith, Albert Reese • PAG/VHOs are public charities with active fundraising components that work to improve health by supporting advocacy, education & training, health services, and/or research.

4. 2011: LANDSCAPE & TRENDS • Ongoing federal and state budget shortfalls • Retrenchment of pharmaceutical industry R&D • Increasing globalization & expensive U.S. clinical trials • Difficulty recruiting U.S. patients for clinical trials • Inadequate pipeline of clinical researchers & insufficient career paths • Poor harmonization of requirements leading to increased regulatory burden • Responding to these challenges requires increased collaboration across all sectors.

5. Role of PAG/VHOs in Clinical Research • Substantial political clout & credible advocates • Source of community education & outreach • Research funders • Access to special patient groups, registries & samples • Bridge to industry • A paradigm shift is occurring, with PAG/VHOs playing greater roles in setting the clinical research agenda in many disease areas.

6. Estimated 2010 U.S. Health Research Expenditures Data from Research!America • PAG/VHOs provide only a small portion of total U.S. health research expenditures.

7. PAG/VHO Sector: Size & Diversity Well over 10,000 organizations with local, national, or international reach Annual budgets range from less than $100K to well over $1 billion The activities of these organizations and the approaches they use range widely.

8. PAG/VHO Sector: Types of Organizations Traditional PAG/VHOs (support broad range of activities including research & training to help build fields): March of Dimes, American Cancer Society, American Heart Association, the Arthritis Foundation Entrepreneurial (venture) PAG/VHOs(focus primarily on developing products): Multiple Myeloma Foundation, Myelin Repair Foundation, Cystic Fibrosis Foundation Groups using mixed approaches: Juvenile Diabetes Foundation, Susan G. Komen for the Cure • Sector is very heterogeneous—one of its great strengths.

9. Strengths of PAG/VHO Sector • Number & diversity of organizations • Flexibility & ability to move quickly • Laser focus on specific patient interests • When needed, willingness to work on tough problems & make long-term investments • Freedom from for-profit & political pressures • Breadth of activities supported: Funding research, patient care, education, and/or advocacy

10. Umbrella Organizations • National Health Council • Health Research Alliance • Research!America • National Organization for Rare Disorders • Genetic Alliance • Faster Cures • A number of umbrella groups exist, but no single group represents a majority of PAG/VHOs. Nonetheless, such groups are potential partners for the CRForum and AHCs.

11. Government Programs Foster Partnerships with PAG/VHOs Examples include: • Office of Rare Disease Research • Supports consortia that foster collaboration between the rare-diseases community & AHCs • Clinical & Translational Science Award Program • Requires community engagement

12. Examples of Productive Partnerships Between PAG/VHOs & AHCs:Supporting Career Development & Training • American Heart Association (AHA) • Supports early career development &mentoring programs • Currently funds >2,000 researchers • American Society of Clinical Oncology (ASCO) • Offerings include loan repayment program & awards for investigators in developing countries

13. Examples of Productive Partnerships: Creating Research Networks to Foster Data Sharing &Interactions with Industry • Cystic Fibrosis Foundation • Funded network of AHC research hubs with strong emphasis on developing drug pipeline of for CF patients • Multiple Myeloma Research Foundation • Created collaborative research network linked to industry to launch clinical trials & accelerate drug development

14. Examples of Productive Partnerships: Creating Centralized Resources for Rare-disease Research • Many researchers at AHCs use patient registries, shared databases & tissue banks provided by PAG/VHOs to study patients with rare diseases. PAG/VHOs offering such resources include: • Progeria Research • Pseudoxanthoma Elasticum International • Lymphangioleiomyomatosis Foundation

15. Examples of Productive Partnerships:Developing Biomarkers & Diagnostics • Alzheimer’s Association • WorldWide Alzheimer’s Disease Neuroimaging Initiative

16. Examples of Productive Partnerships:Training Volunteers to Become Research Advocates • Susan G. Komen for the Cure • Created a community of advocates to participate in a variety of scientific activities, such as grant review & advocacy.

17. Update on CRForum Initiative: Partnering with PAG/VHOs • Activities: Outreach to PAG/VHO community to identify issues of concern & share information with AHCs • Phone & in-person interviews • Panels at annual CRForum meetings • Web survey designed with input from the Health Research Alliance and Genetic Alliance and sent to their members • Products: white paper & related manuscript for publication, as well as this slide presentation

18. Feedback from PAG/VHOs: Results of Phone Interviews & Meetings • In winter and spring of 2010, QE Philanthropic Advisors conducted: • Phone interviews with ~10 PAG/VHOs & 2 foundations • In-person discussion with 3 umbrella groups

19. Feedback from PAG/VHOs: Results of Phone Interviews & Meetings • Contracting processes are cumbersome and often take too long. • Communication between PIs and contracting offices is often inadequate. • Many PAG/VHOs want “return on investment” and include requirements for profit sharing, IP sharing& “reach-through clauses” in their contracts; this prolongs negotiations.

20. Feedback from PAG/VHOs: Formal Web Survey (Summer 2011) • Designed with Health Research Alliance (HRA) & Genetic Alliance; distributed to their members • Survey responses collected 8/2011 to 9/2011 • 37 respondents • 27 PAG/VHOs; 20 focused on single disease or disease area • 10 foundations

21. Feedback from PAG/VHOs: Formal Web Survey • Out of 27 PAG/VHOs: • Total 2010 budgets ranged from $0.2-$272 M • 20 focused on single disease or disease area • 23 funded health research • % of clinical health research funded in 2010 ranged from 25%-100% • 9/23 supported development of young investigators

22. Survey Questions : What is your organization’s total budget for 2010? What is your organization’s total health research budget for 2010? • The percent budget spent on health research by the 23 responding PAG/VHOs varied from ~9% to 90%.

23. Survey Question: What % of research supported by your organization is clinical and/or translational research? % Responding VHOs • Percentage of total research funded in 2010 that was clinical and/or translational ranged widely for the 23 PAG/VHOs responding to the survey.

24. Survey Request: For each area of work, indicate how much of the work your organization funds (whether conducted directly by your organization or through indirect grants or contracts to other organizations) is carried out with AHCs. • 83% of respondents indicated ≥50% of research they support is carried out with AHCs, while only 23% of respondents carry out most of their capacity- building and training activities with AHCs.

25. Survey Request: For each area of work, indicate how much of the work your organization funds (whether conducted directly by your organization or through indirect grants or contracts to other organizations) is carried out with AHCs. • Only a few organizations carried out most of their activities relating to patient registries, patient services & advocacy with AHCs.

26. Survey Request: Assess the quality of interactions between your organization & AHCs for each area of work supported by your organization. • For research & capacity building/training activities, respondents indicated their interactions with AHCs were consistently or usually excellent.

27. Survey Request: Assess the quality of interactions between your organization & AHCs for each area of work supported by your organization. • For non-research and training activities, respondents’ assessments of the quality of their interactions with AHCs were mixed.

28. Survey Question: Have any of the factors listed below significantly slowed down and/or negatively impacted your interactions with AHCs? • Most respondents identified bureaucratic processes, negotiating intellectual property & IRB delays as often problematic.

29. What Can/Should AHCs Do Now? • Improve communications and linkages. • Provide timely feedback from clinical trials to patients & PAG/VHOs. • Seek feedback and advice from patients and PAG/VHOs on research required to address patient needs, and the experiences of clinical trial participants. • Work with PAG/VHOs to establish educational processes that systematically reach out to, support& educate for these roles.

30. What Can/Should AHCs Do Now? • Partner with PAG/VHOs using existing PAG/VHO educationalmaterials and community programs. • Conduct systems/engineering process exercises to streamline bureaucratic processes. • Work with PAG/VHOs supporting career development programs to develop career paths and incentives to retain clinician-scientists.

31. What Can/Should AHCs Do Now? • Engage PAG/VHOs in helping simplify informed consent documents. • Explore ways to help PAG/VHOs recruit investigators to study the diseases on which they focus by developing careers of physician-scientists & building fields of research.

32. Summary & Conclusions • Partnerships & collaborations can bridge different parts of the clinical research enterprise and facilitate translation &application of new knowledge. • Such partnerships and collaborations can help AHCs innovate to enhance organizational effectiveness &achieve their goals.

33. Summary & Conclusions • PAG/VHOs are natural partners for AHCs: both have a deep commitment to improving health for their communities. • PAG/VHOs not only provide research funds, but also help recruit patients to trials, link to industry, catalyze innovation, & support cross-sector research networks to facilitate data sharing &speed product development. • PAG/VHOs strengths should be fully leveraged by AHCs and the nation as a whole.